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Scientists from the NIHR Manchester Biomedical Research
Centre (BRC) say a naturally occurring chemical found in soy could
prove to be an effective new treatment for a fatal genetic disease
that affects children.

Dr Brian Bigger, from the BRC's MPS Stem Cell Research
Laboratory, found that genistein - derived from soya beans and
licensed in the US as an osteoporosis drug - had a dramatic effect
on mice suffering from the human childhood disease Sanfilippo.

"Sanfilippo is an untreatable mucopolysaccharide (MPS) disease
affecting one in 89,000 children in the United Kingdom," said Dr
Bigger, who is based in Saint Mary's Hospital and The University of
Manchester's School of Biomedicine.

"Children with Sanfilippo disease experience progressive
deterioration of mental function, similar to dementia, in early
childhood, with other symptoms including severe behavioural
problems, hyperactivity and ultimately death in early teens."

In the study, published in the journal Public Library of Science
One, mice with Sanfilippo disease were fed with high doses of
genistein over a nine-month period. Treated mice showed a
significant delay in their mental decline, including a third
reduction in the amount of excess sugars found in the brain as a
result of the disease, and a sixth reduction in inflammation in the
brain.

Importantly, the research also showed that the hyperactivity and
other abnormal behaviour normally seen in Sanfilippo mice were
fully corrected by genistein treatment.

Professor Ed Wraith, a co-author on the study and consultant
paediatrician from Genetic Medicine within Saint Mary's Hospital,
said "Sanfilippo is a disease where the genetic lack of an enzyme
leads to a fault in the breakdown of complex sugars in the
cell.

"This leads to storage of these undegraded complex sugars in
cells, disturbances in brain function and ultimately to this
profound mental deterioration that we see in the children with this
condition. Manchester is a specialist centre for this type of
genetic disease and as such we look after more than 100 patients
from all over the UK and beyond."

The Manchester team, supported by the UK society for
mucopolysaccharide diseases, hope to announce a placebo controlled
clinical trial for patients with Sanfilippo disease in the near
future.

EndsNotes to editors:
A copy of the paper is available on request.
More information can be found at:

The NIHR Manchester Biomedical Research Centre was created by
the National Institute for Health Research in 2008 to effectively
move scientific breakthroughs from the laboratory, through clinical
trials and into practice within hospitals to improve patient care.
As a partnership between Central Manchester University Hospitals
NHS Foundation Trust and The University of Manchester, the
Biomedical Research Centre is designated as a specialist centre of
excellence in genetics and developmental medicine. www.manchesterbrc.org